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Monoclonal Antibodies

Burosumab for CSHS

Phase < 1

Waitlist Available

Led By Laura Tosi, MD

Research Sponsored by Laura Tosi

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 52 weeks

Awards & highlights

No Placebo-Only Group

Summary

This trial is testing whether the drug burosumab can help patients with Cutaneous Skeletal Hypophosphatemia Syndrome by reducing the levels of a protein called fibroblast growth factor 23.

Eligible Conditions

Rickets
Epidermal Nevus Syndrome

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 52 weeks

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~52 weeks

This trial's timeline: 3 weeks for screening, Varies for treatment, and 52 weeks for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Serum Phosphorus change

Secondary study objectives

6-minute walk test

Biomechanical Marker

Brief Fatigue Inventory (BFI)

+10 more

Side effects data

From 2021 Phase 2 trial • 17 Patients • NCT02304367

64%

Pain In Extremity

64%

Arthralgia

50%

Upper Respiratory Tract Infection

50%

Cough

43%

Diarrhoea

36%

Nausea

36%

Vomiting

36%

Nasopharyngitis

36%

Back Pain

36%

Muscle Spasms

29%

Constipation

29%

Neoplasm Progression

29%

Urinary Tract Infection

29%

Musculoskeletal Pain

29%

Insomnia

29%

Hypertension

29%

Pain

29%

Fall

29%

Myalgia

29%

Amylase Increased

21%

Contusion

21%

Abdominal Pain

21%

Oedema Peripheral

21%

Influenza

21%

Ligament Sprain

21%

Procedural Pain

21%

Hypomagnesaemia

21%

Vitamin D Deficiency

21%

Joint Range Of Motion Decreased

21%

Musculoskeletal Chest Pain

21%

Restless Legs Syndrome

21%

Nasal Congestion

21%

Oropharyngeal Pain

21%

Pulmonary Mass

21%

Rash

21%

Fatigue

21%

Respiratory Tract Congestion

21%

Bronchitis

14%

Cardiac Arrest

14%

Tinnitus

14%

Acute Respiratory Failure

14%

Anaemia

14%

Abdominal Pain Lower

14%

Toothache

14%

Asthenia

14%

Injection Site Reaction

14%

Pyrexia

14%

Foot Fracture

14%

Rib Fracture

14%

Tooth Fracture

14%

Creatinine Renal Clearance Decreased

14%

Lipase Increased

14%

Decreased Appetite

14%

Hyperglycaemia

14%

Hyperphosphataemia

14%

Hypocalcaemia

14%

Hypokalaemia

14%

Bone Lesion

14%

Bone Pain

14%

Anxiety

14%

Depression

14%

Nephrolithiasis

14%

Dyspnoea

14%

Upper Respiratory Tract Congestion

14%

Hypotension

14%

Chills

14%

Exostosis

14%

Neck Pain

14%

Dizziness

14%

Headache

14%

Acute Kidney Injury

14%

Abdominal Discomfort

14%

Abdominal Distension

14%

Joint Swelling

Concussion

Abdominal Pain Upper

Ascites

Blood Creatinine Increased

Hyperuricaemia

Melanocytic Naevus

Poor Quality Sleep

Bile Duct Stone

Hypoalbuminaemia

Hepatic Lesion

Blood Creatine Increased

Seasonal Allergy

Hepatic Cyst

Basal Cell Carcinoma

Dental Caries

Metastases To Pancreas

Renal Impairment

Echocardiogram Abnormal

Bradycardia

Retching

Pneumonia

Multi-Organ Failure

Pulseless Electrical Activity

Tooth Abscess

Cholangitis

Sialoadenitis

Eyelid Ptosis

Rheumatoid Arthritis

Metastases To Lung

Ovarian Cancer

Tumour Compression

Pickwickian Syndrome

Pulmonary Embolism

Coagulopathy

Leukocytosis

Tachycardia

Abdominal Mass

Dysphagia

Flatulence

Gastrooesophageal Reflux Disease

Hypoaesthesia Oral

Large Intestine Polyp

Localised Intraabdominal Fluid Collection

Poor Dental Condition

Stomatitis

Application Site Rash

Calcinosis

Chest Pain

Generalised Oedema

Injection Site Bruising

Injection Site Swelling

Medical Device Site Reaction

Nodule

Cystitis

Diverticulitis

H1n1 Influenza

Oesophageal Candidiasis

Oral Candidiasis

Sinusitis

Viral Upper Respiratory Tract Infection

Hip Fracture

Ligament Rupture

Tooth Injury

Blood Calcium Increased

Blood Cholesterol Increased

Blood Glucose Increased

Blood Parathyroid Hormone Increased

Blood Potassium Decreased

Blood Pressure Systolic Increased

Blood Sodium Decreased

Blood Uric Acid Increased

Computerised Tomogram Abdomen Abnormal

Electrocardiogram Abnormal

Electrocardiogram Qt Prolonged

Glycosylated Haemoglobin Increased

Liver Function Test Abnormal

Oxygen Saturation Decreased

Waist Circumference Increased

White Blood Cell Count Decreased

Gout

Hypercalcaemia

Hyponatraemia

Hypophosphataemia

Hypoproteinaemia

Metabolic Acidosis

Aneurysmal Bone Cyst

Bursitis

Flank Pain

Intervertebral Disc Annular Tear

Joint Stiffness

Muscle Mass

Osteosclerosis

Neuroma

Squamous Cell Carcinoma Of Skin

Thyroid Neoplasm

Cerebral Cyst

Clonus

Sensory Disturbance

Viith Nerve Paralysis

Agitation

Panic Attack

Sleep Talking

Haematuria

Hydronephrosis

Micturition Urgency

Nephrocalcinosis

Pollakiuria

Proteinuria

Renal Cyst

Urinary Incontinence

Prostatomegaly

Testicular Mass

Vaginal Haemorrhage

Haemothorax

Pulmonary Artery Dilatation

Actinic Keratosis

Decubitus Ulcer

Keloid Scar

Precancerous Skin Lesion

Pruritus

Rash Papular

Skin Irritation

Skin Lesion

Skin Mass

Skin Odour Abnormal

Deep Vein Thrombosis

Peripheral Venous Disease

Vasodilatation

Road Traffic Accident

Calculus Bladder

Pancreatic Carcinoma Metastatic

Pleural Effusion

Pancreatitis

Dyspepsia

Gait Disturbance

Peripheral Swelling

Gastrointestinal Infection

Gastrointestinal Viral Infection

Laceration

Post Procedural Haematoma

Scar

Skin Abrasion

Spinal Compression Fracture

Stress Fracture

Tibia Fracture

Ultrasound Kidney Abnormal

Hypercholesterolaemia

Soft Tissue Mass

Tendon Pain

Schwannoma

Squamous Cell Carcinoma

Dysgeusia

Hypoaesthesia

Sciatica

Vulva Cyst

Respiratory Acidosis

Septic Shock

Abdominal Hernia

Injection Site Pain

Conjunctivitis

Corona Virus Infection

Femur Fracture

Fibula Fracture

Radiation Mucositis

Musculoskeletal Stiffness

Migraine

Neuralgia

Paraesthesia

Chylothorax

Prostatic Specific Antigen Increased

100%

80%

60%

40%

20%

Study treatment Arm

KRN23 TIO

KRN23 XLH and ENS

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups

Experimental Treatment

Group I: BurosumabExperimental Treatment1 Intervention

Burosumab, which is FDA-approved for X-linked hypophosphatemic rickets, will be given monthly, for a total of 12 months and titrated to achieve a target fasting serum phosphorus level within normal range for age. The chosen starting dose of burosumab will be 0.3 mg/kg given SQ Q4W. The maximum dose allowed in this protocol is 2.0 mg/kg. Burosumab will be administered via subcutaneous (SC) route.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Burosumab

2021

Completed Phase 3

~120

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